DDrare: Database of Drug Development for Rare Diseases

285. ファンコニ貧血
［臨床試験数：56，薬物数：111（DrugBank：31），標的遺伝子数：30，標的パスウェイ数：151］

Searched query = "Fanconi anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = AMD3100; AP1903; AP1903 A594; ATG; Abatacept; Alefacept; Anti-CD45; Anti-Thymocyte Globulin; Anti-Thymocyte Globulin (Rabbit); Anti-thymocyte globulin; BPX-501; BPX-501 T cells; BPX-501 cells; Busulfan; Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.; CAMPATH-1H; CD34+ CELLS; CD34+ Cells; CD34+ selected cells; CD34+CELLS; Campath; Campath 1h; Chemotherapy; Cyclophosphamide; Cyclophosphamide (CY) (Plan 1); Cyclophosphamide (CY) (Plan 2); Cyclophosphamide 30; Cyclophosphamide 40; Cyclophosphamide, Fludarabine; Cyclosporine; Danazol; Device: AmCell CliniMACs; Device: CliniMACS device; Device: CliniMACs device; Device: Donor mobilized PBSC infusion; Device: The CliniMACS device; ENBREL*SC 4FL 25MG+4SIR 1ML; Eltrombopag; Enbrel; Etanercept; FP-045; Filgrastim; Fludarabine; Fludarabine (FLU); Fludarabine Phosphate; Fludarabine phosphate; G-CSF; Genetically Engineered Hematopoietic Stem Progenitor Cells; Genetically Engineered Hematopoietic Stem/Progenitor Cells; Graft-versus-tumor induction therapy; Hematopoietic stem cell transplantation; Melphalan; Metformin; Metformin HCl; Methotrexate; Methylprednisolone; Methylprednisolone (MP); Miltenyi CliniMACS; Mozobil; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Neupogen; Olaparib; Other: Laboratory Biomarker Analysis; Oxandrolone; Pembrolizumab; Peripheral blood stem cell; Phosphate; Plerixafor; Plérixafor; Prednisone; Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Transplantation; Procedure: CD34 selected haploidentical PBSCT; Procedure: Hematopoietic Stem Cell Transplant; Procedure: Hematopoietic Stem Cell Transplantation; Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs); Procedure: Leukapheresis; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Stem cell infusion; Procedure: Thymic Shielding During Radiation; Procedure: Total Body Irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation; Quercetin; Quercetin (dietary supplement); RP-L102; Rabbit; Rabbit ATG; Radiation: Total body irradiation (TBI); Radiation: Total-Body Irradiation; Radiation: radiation therapy; Radiation: total-body irradiation; Rimiducid; Rituximab; Rivogenlecleucel; Rivogenleucleucel; Sargramostim; Sirolimus; T cell infusion; Tacrolimus; Therapeutic allogeneic lymphocytes; Total Body Irradiation (TBI) (Plan 1); Transduced CD34+ Cells; Zarzio

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT03579875 (ClinicalTrials.gov)	November 13, 2018	25/5/2018	T Cell Receptor a/ß TCD HCT in Patients With Fanconi Anemia	T Cell Receptor Alpha/Beta T Cell Depleted (a/ß TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA)	Fanconi Anemia;Severe Aplastic Anemia;Myelodysplastic Syndromes	Drug: Total Body Irradiation (TBI) (Plan 1);Drug: Cyclophosphamide (CY) (Plan 1);Drug: Fludarabine (FLU);Drug: Methylprednisolone (MP);Device: Donor mobilized PBSC infusion;Drug: G-CSF;Drug: Cyclophosphamide (CY) (Plan 2);Drug: Rituximab;Drug: Busulfan	Masonic Cancer Center, University of Minnesota	NULL	Recruiting	N/A	65 Years	All	48	Phase 2	United States
2	NCT03600909 (ClinicalTrials.gov)	May 15, 2018	17/7/2018	A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia	A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine	Fanconi Anemia;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML)	Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Anti-Thymocyte Globulin (Rabbit);Device: The CliniMACS device;Drug: G-CSF	Memorial Sloan Kettering Cancer Center	Pediatric Brain Tumor Consortium	Recruiting	1 Month	N/A	All	70	Phase 2	United States
3	NCT02678533 (ClinicalTrials.gov)	February 10, 2017	5/2/2016	Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor	Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy	Fanconi Anemia	Drug: G-CSF;Drug: Plerixafor	Assistance Publique - Hôpitaux de Paris	EuroFancolen	Recruiting	2 Years	17 Years	All	8	Phase 1;Phase 2	France
4	NCT02143830 (ClinicalTrials.gov)	April 2014	28/4/2014	HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy	A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine	Fanconi Anemia;Severe Marrow Failure;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML)	Drug: Busulfan;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: rabbit ATG;Drug: G-CSF;Biological: Peripheral blood stem cell	Children's Hospital Medical Center, Cincinnati	Memorial Sloan Kettering Cancer Center;Fred Hutchinson Cancer Research Center	Recruiting	3 Months	N/A	All	70	Phase 2	United States
5	NCT00479115 (ClinicalTrials.gov)	May 2007	23/5/2007	Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100	AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II Study	Fanconi Anemia	Drug: AMD3100;Device: AmCell CliniMACs	Children's Hospital Medical Center, Cincinnati	National Heart, Lung, and Blood Institute (NHLBI)	Completed	1 Year	30 Years	All	1	Phase 1;Phase 2	United States
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
6	EUCTR2014-005264-14-FR (EUCTR)		19/06/2015	FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemia	NA - EUROFANCOLEN	Fanconi Anemia MedDRA version: 18.1;Level: LLT;Classification code 10055206;Term: Fanconi's anemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Zarzio Product Name: Zarzio INN or Proposed INN: filgrastim Other descriptive name: G-CSF Trade Name: Mozobil Product Name: Mozobil INN or Proposed INN: plérixafor	ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)	NULL	NA			Female: yes Male: yes		Phase 2	France

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT03579875
(ClinicalTrials.gov)

November 13, 2018

25/5/2018

T Cell Receptor a/ß TCD HCT in Patients With Fanconi Anemia

T Cell Receptor Alpha/Beta T Cell Depleted (a/ß TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA)

Fanconi Anemia;Severe Aplastic Anemia;Myelodysplastic Syndromes

Drug: Total Body Irradiation (TBI) (Plan 1);Drug: Cyclophosphamide (CY) (Plan 1);Drug: Fludarabine (FLU);Drug: Methylprednisolone (MP);Device: Donor mobilized PBSC infusion;Drug: G-CSF;Drug: Cyclophosphamide (CY) (Plan 2);Drug: Rituximab;Drug: Busulfan

Masonic Cancer Center, University of Minnesota

NULL

Recruiting

N/A

65 Years

All

Phase 2

United States

NCT03600909
(ClinicalTrials.gov)

May 15, 2018

17/7/2018

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Fanconi Anemia;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML)

Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Anti-Thymocyte Globulin (Rabbit);Device: The CliniMACS device;Drug: G-CSF

Memorial Sloan Kettering Cancer Center

Pediatric Brain Tumor Consortium

Recruiting

1 Month

N/A

All

Phase 2

United States

NCT02678533
(ClinicalTrials.gov)

February 10, 2017

5/2/2016

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor

Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy

Fanconi Anemia

Drug: G-CSF;Drug: Plerixafor

Assistance Publique - Hôpitaux de Paris

EuroFancolen

Recruiting

2 Years

17 Years

All

Phase 1;Phase 2

France

NCT02143830
(ClinicalTrials.gov)

April 2014

28/4/2014

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Fanconi Anemia;Severe Marrow Failure;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML)

Drug: Busulfan;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: rabbit ATG;Drug: G-CSF;Biological: Peripheral blood stem cell

Children's Hospital Medical Center, Cincinnati

Memorial Sloan Kettering Cancer Center;Fred Hutchinson Cancer Research Center

Recruiting

3 Months

N/A

All

Phase 2

United States

NCT00479115
(ClinicalTrials.gov)

May 2007

23/5/2007

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100

AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II Study

Fanconi Anemia

Drug: AMD3100;Device: AmCell CliniMACs

Children's Hospital Medical Center, Cincinnati

National Heart, Lung, and Blood Institute (NHLBI)

Completed

1 Year

30 Years

All

Phase 1;Phase 2

United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2014-005264-14-FR
(EUCTR)

19/06/2015

FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemia

NA - EUROFANCOLEN

Fanconi Anemia
MedDRA version: 18.1;Level: LLT;Classification code 10055206;Term: Fanconi's anemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: Zarzio
Product Name: Zarzio
INN or Proposed INN: filgrastim
Other descriptive name: G-CSF
Trade Name: Mozobil
Product Name: Mozobil
INN or Proposed INN: plérixafor

ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)

NULL

Female: yes
Male: yes

Phase 2

France